Access to Immuno-Oncology Therapies - European Policy Perspective

Suzanne Wait, PhD

Ensuring rapid, appropriate, and sustainable access to immuno-oncology therapies for patients in Europe: what role can policies play?

Immuno-oncology (I-O) therapies are expected to transform the treatment landscape for many forms of advanced cancer in years to come. The pace of development of these agents is remarkable, with multiple agents targeting different immune mechanisms being investigated for the treatment of some of the most common and most difficult-to-treat cancers.1

Understandably, patient groups are urging policymakers to make these innovative treatments available to patients as quickly as possible. However, the price tag for these new drugs is high, and policymakers have to find ways to accommodate these new agents within financially constrained healthcare budgets.2

It was in light of these political realities that the European Expert Group on Immuno-Oncology, an independent network of patient representatives, cancer clinical experts, scientists, and policymakers, issued the Policy Action Framework for Immuno-Oncology in 2014,3 with the aim to guide policymakers toward concrete steps that may create an enabling policy environment for rapid and appropriate access to I-O drugs across Europe.4 The Framework proposed 5 concrete steps that policymakers may take to foster access to I-O treatments (Figure 1). One year after its publication, we look at how some of these proposals have unfolded across Europe.

More Flexible, and More Efficient, Regulatory and Access Pathways

The science of I-O is continually evolving, and experts have called for adaptation to clinical development pathways5 and trial endpoints6,7 to best reflect the clinical patterns observed with I-O agents and capture their full benefits.8 Experts have also called for greater flexibility in regulatory pathways for promising new cancer drugs, including I-O agents, to ensure they are made available to patients as quickly as possible.9 With this goal in mind, the European Medicines Agency (EMA) is piloting a number of adaptive licensing schemes, which allow approval decisions to be based on an evolving set of data for drugs that show promising results in early phase clinical trials, with an ultimate aim to make them available to patients more quickly.10,11

Accelerating the drug approval process, however, is only one part of the access equation. In Europe, although drug approval is centralized with the EMA, each country maintains jurisdiction over the financing of its healthcare system. Access frameworks vary considerably across the member states, with some including a formal health technology assessment (HTA) and others relying on pricing and reimbursement mechanisms. Each access process also has its own evidentiary requirements. As a result, there are significant differences in the time it takes new drugs approved by the EMA to become available to patients in different European countries.12

Figure 1. Recommendations of the Action Framework on Immuno-Oncology

These disparities in access have drawn considerable attention from patient groups and politicians alike.13 In 2012, a European Commission Directive reduced the limit for the duration of pricing and reimbursement processes in member states from 180 days to 120 days.14 The European Network for Health Technology Assessment has also tried to harmonize evidence requirements for new drugs across countries.

Despite these efforts, significant delays in access still remain in many countries, particularly in Eastern Europe.12 This is illustrated in the case of trastuzumab for the treatment of metastatic cancer in Figure 2.15

National-level reimbursement or coverage is critical; however, it does not necessarily result in patients having access to new drugs, as funding decisions are increasingly decentralized to the regional and sometimes the individual hospital level.2 Inequities in access to all aspects of cancer care are widespread and are potentially increasing with the recent austerity measures introduced in a number of European countries.13 Out-of-pocket payments are also increasing in light of limited public funding for new drugs in many countries, posing important ethical questions for societies whose healthcare systems are historically based on principles of solidarity and universal access.